Join Sparks, Novartis, bluebird bio, UniQure, Pfizer, Shire, Nightstar, Vertex, AvroBio & many more at the 9th World Orphan Drug Congress!

Dear Victor Norberto, The 9th World Orphan Drug Congress is taking place 6 th – 8th November 2018 inBarcelona, Spain! Our 2018 agenda focuses on 2 days’ worth of sessions covering the full breadth of the rare disease space, and this year we’ve expanded the Cell & Gene Therapyfocus. KEY THEMES: Clinical Advancements in Cell & Gene Therapy

Nerissa C. Kreher MD, MBA, CMO AvroBio   Dr Jerome Rossert VP, General Medicine, Global Clinical Development Vertex Pharmaceuticals   Dr Greg Robinson CSO Nightstar Therapeutics

The realities of implementing gene therapy Creating pipelines that are robust enough for gene therapy to become a reality Preparing for later stage trials & commercialization

Dr Sander van Deventer CSO UniQure   Dr Tuyen Ong CDO Nightstar Therapeutics   Karen Aiach Founder and CEO Lysogene   Dr Michael Linden Former VP Gene Therapy & Head GMI

Overcoming manufacturing hurdles  How can gene therapy companies make their manufacturing process more cost effective and sustainable? Industrialization of gene and cell therapies manufacturing Post approval: Next steps to the production process

Dr Frederic Revah CEO, Genethon & President Yposkesi   Dr Emanuele Ostuni Head of Europe, Cell and Gene Therapy Novartis Oncology Region Europe   Marian Bendik Head of Gene Therapy, Process Development & Technical Services Shire   James Christie SVP Manufacturing & Supply Chain MeiraGTx

Pricing & Reimbursement  Innovative payment models for cell & gene therapies

Agnese Cangini Italian Medicine Agency AIFA   Dr Alexander Natz Secretary General EUCOPE   Sarah Pitluck Head, Global Pricing & Reimbursement Spark Therapeutics

Dr Yvonne Schmidt Scientific Advisor, Pharmaceutical Department Federal Joint Committee (G-BA), Germany   Dr Daniela Settesoldi Technical Scientific Committee and Pricing and Reimbursement Committee, Italian Medicine Agency AIFA    Leo Strican Head of Access Value & Evidence Strategy, Europe bluebird bio

VIEW THE 2018 AGENDA We expect over 450 attendees. This is your best chance of the year to meet and network with hundreds of like-minded peers.  See you in Barcelona! Best wishes, Wing-yun Cheung Project Director World Orphan Drug Congress Email: wing-yun.cheung@terrapinn.com Tel: +44 20 7092 1172 Our Advisory Board

Yann Le Cam, Chief Executive Officer, EURORDIS   Dr Alexander Natz, Secretary General, EUCOPE   Dr Bruno Sepodes, Chair of COMP, EMA   Alastair Kent, Former Director, Genetic Alliance UK

Professor Michael Linden, Former VP Gene Therapy & Head, GMI, Pfizer    Wills Hughes-Wilson, Head of Patient Access & Commercial Planning, Mereo BioPharma   Dr Ségolène Aymé, Founder of Orphanet, Emeritus Research Director, INSERM   Sheela Upadhyaya, Associate Director Highly Specialised Technologies, NICE

Dr Carlo Incerti, SVP, Head of Global Medical Affairs, CMO, Genzyme, A Sanofi Company   Dr Mathew Pletcher, Head of Rare Disease Discovery, Roche    Dr M Ken Kengatharan, Managing Director, Atheneos   Rick Thompson, CEO, Findacure

Dr Birgitte Volck, SVP, Head of R&D Rare Diseases, GSK   Dr Pedro Franco, Former Scientific Administrator, EMA   Anna Bucsics, Project Advisor, MoCA   Josie Godfrey, Former Associate Director, NICE